Clinical trials: Severe Hemophilia A, open to volunteers
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Severe Hemophilia A Without Inhibitor
A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
Study Objectives* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3.
- Trial name
- NCT05981274
- Testing
- serum biomarker.
- Where
- Taiwan
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Tri-Service General Hospital (TSGH)
Hemophilia A Patient
Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly…
- Trial name
- E-PROTECT (NCT07830693)
- Testing
- Study questionnaires, Clinical/pharmacological data collection
- Where
- 8 sites in France
- Volunteers
- 553 people
- Sponsor
- Nantes University Hospital
Hemophilia A, Hemophilia A, Severe
Hemophilia A Research Program
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood.
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency
- Trial name
- HARP (NCT07414511)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Washington
Hemophilia A
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process.
- Trial name
- STEP (NCT05022459)
- Testing
- Emicizumab, FVIII
- Where
- 10 sites in United States
- Volunteers
- 72 people
- Sponsor
- Wayne State University
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia A, Hemophilia B
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study…
- Trial name
- NCT05145127
- Testing
- PF-06741086
- Where
- 78 sites in United States, Argentina and 22 more
- Volunteers
- 245 people
- Sponsor
- Pfizer
Hemophilia A
Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis
Patients with severe hemophilia A often develop joint complication requiring careful monitoring. This study aimed to compare the effectiveness of standard low dose weight-based FVIII concentrates (CFCs) prophylaxis with…
- Trial name
- HEADUS_SHA8 (NCT06816953)
- Testing
- HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen, Inflammatory markers
- Where
- Thailand
- Volunteers
- 19 people
- Sponsor
- Chulalongkorn University
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Severe Hemophilia A
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
This study was divided into four stages: screening period, main trial period, extension period and follow-up period. In the main trial, both groups received FRSW107 prophylactic therapy.
- Trial name
- NCT06136507
- Testing
- FRSW107
- Where
- 13 sites in China
- Volunteers
- 76 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Von Willebrand Disease, Type 3, Concomitant VWD and Hemophilia
Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges.
- Trial name
- BCDI-XII (NCT05500807)
- Testing
- Emicizumab
- Where
- 12 sites in United States
- Volunteers
- 40 people
- Sponsor
- Bleeding and Clotting Disorders Institute Peoria, Illinois
Hemophilia A, Severe
An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
This is a prospective, multicenter, open-label study to assess efficacy, safety, pharmacokinetics (PK), and immunogenicity of human plasma-derived Factor VIII (FVIII) in previously treated patients (PTPs) with severe hemophilia…
- Trial name
- NCT06738901
- Testing
- SKP-0141
- Volunteers
- 55 people
- Sponsor
- SK Plasma Co., Ltd.
Hemophilia A
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts: Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia…
- Trial name
- NCT05987449
- Testing
- NXT007
- Where
- 14 sites in United States, Canada and 4 more
- Volunteers
- 60 people
- Sponsor
- Hoffmann-La Roche
Severe Hemophilia A
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors.
- Trial name
- NCT06703606
- Testing
- MARSTACIMAB
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Hemophilia A, Factor VIII (FVIII)
TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies
This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they…
- Trial name
- NCT07692217
- Testing
- Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of Texas Southwestern Medical Center
Severe Hemophilia A
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis
- Trial name
- NuPOWER (NCT05935358)
- Testing
- Nuwiq
- Where
- 18 sites in United States, Croatia and 9 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
Severe Hemophilia A, Severe Hemophilia A With Inhibitor
Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
The goal of this observational study is to learn about the changes of antibodies and inhibitors against the coagulation factor VIII in patients with severe hemophilia A receiving emicizumab therapy.
- Conditions
- Severe Hemophilia A, Severe Hemophilia A With Inhibitor, Severe Hemophilia A Without Inhibitor
- Trial name
- NAVIGATE (NCT05802836)
- Testing
- no interventions
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- Christoph Königs
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals